Even though cancer is responsible for more deaths in children than all other diseases combined, the road to developing new treatments for pediatric cancer is long and slow. There are roughly four phases in the development of new therapies: basic research, translational research, clinical trials, and FDA review and approval.
Basic research is motivated by scientific curiosity without a specific end goal or product in mind. In contrast to applied research, which focuses on the practical use of scientific discoveries, basic research is undertaken for the advance of scientific knowledge and often serves as a foundation for the applied work that follows. One example of basic research is the development of recombinant-DNA techniques in the laboratory. This discovery has allowed scientists to study human genetics in much greater detail, so that scientists can now understand the precise coding of cancer mutations.
The second phase of research, translational research, takes discoveries in basic science and tries to apply them to treating and preventing diseases. This phase of research has been nicknamed the “valley of death” because it is incredibly difficult to move a discovery successfully from basic science to clinical application. Sometimes, researchers will test a compound unsuccessfully on one disease and find years later that this same compound is effective for treating a completely different disease. This is true for raloxifene, a drug developed for osteoporosis that is actually more effective at breast cancer prevention. Translational research includes identifying cancer biomarkers and targets, and developing early testing in animal models. Even though translational research is often not successful, it is the crucial bridge between early discoveries and new drugs for patients.
Once researchers have found compounds that work on animal models, they move into the third phase of research: clinical trials. Clinical trials test new treatments on human patients. Clinical trials also have phases, which you can read more about here. Once a new treatment has gone through clinical trials, it must also go through FDA review and approval, another lengthy process in which the FDA determines whether the treatment is effective and safe for all patients.
The funding model for the current pipeline is piecemeal, contributing to the slow pace of drug development. The early stages of the pipeline are primarily funded by public sources like the National Institute of Health, while the latter stages of clinical development and FDA review are chiefly funded by for-profit industry like pharmaceuticals. Translational research is often a mix of government funding, private funding, and pharmaceutical development. CureSearch works to accelerate this process by funding research that is moving into clinical development to speed the translation of promising discoveries into potential new therapies to treat children’s cancers. By providing flexible, large-scale funding for researchers with project support and firm research deadlines, CureSearch tries to move translational discoveries into clinical application faster.